A different strategy was found to be far more effective, which involved an experimental drug called LXR-623, an LXR-beta agonist. In an orthotopic GBM mouse model this drug extended mouse survival significantly without toxicity in the healthy brain. By stimulating LXR-beta, the drug suppresses LDL uptake into cells and increases cholesterol efflux from the cell, selectively depriving the GBM cells of cholesterol. The drug is brain-penetrant and has already been in phase 1 safety and pharmacokinetic/pharmacodyamic trial with healthy volunteers.
As a non-approved drug, LXR-623 is not generally available to patients, but we hope to see a trial for GBM initiated based on this excellent preclinical work.
Study abstract: An LXR-Cholesterol Axis Creates a Metabolic Co-Dependency for Brain Cancers (full study will be uploaded to the Library, folder 2)